Prescribed Reading: DMD, MS and other acronyms

Author

Published

Share it

Biopharma is a complex, rapidly evolving industry that is highly regulated and closely watched — and that means there is constant news. Here's a closer look at the clinical trials, M&A, cool science and regulations that are driving the industry this week.

In case you missed it

Mergers & analysis

Big biotech Celgene snapped up some pre-clincial immunology assets this week from Nimbus Therapeutics, adding to its growing pipeline in the space. The tie-up is the culmination of over a year of talks between the two companies and could set up Celgene to later compete with the likes of Bristol-Myers Squibb in the Tyk2 inhibitor space.

Elsewhere, Endocyte pleased shareholders when its inked a pact with German company ABX GmbH, adding a Phase 3-ready prostate cancer asset to its pipeline. The company will now shift its resources to the project and try to out-license the rest of its pipeline —minus a CAR-T asset that will become a secondary focus.

Gilead Sciences Inc. struck a different kind of deal, making a licensing agreement with a United Nations-backed organization to outlicense the manufacturing its yet-to-be-approved HIV medicine for the distribution in 116 low-income nations. Once the drug — which recently reported out solid late-stage results — gets U.S. approval, manufacturers in China, India and South Africa will be able to get it into the hands of low-income patients throughout the world for a cost of as little as $3.50 per patient.

Clinically relevant

Rare disease biotech Amicus Therapeutics reported positive data from an early-stage study in Pompe disease, prompting the company to seek a Phase 3 trial right away and even reach out to the Food and Drug Administration to seek an accelerated approval pathway for the drug. The clinical win largely offset disappointment from investors last month when another of the biotech's drugs performed poorly.

Motif Bio plc is prepping a filing of a New Drug Application for 2018, following the results of a Phase 3 study of its in antibiotic in complex skin infections. While the drug was once rejected by the FDA under a previous owner for not having enough data, the recent study confirms other late-stage results showing the antibiotic works.

Catabasis pleased investors this week, reporting positive results from the open-label extension period of a Phase 2 study in Duchenne muscular dystrophy (DMD) patients. The drug had previously failed to show improvement over placebo in an earlier, blinded part of the study, but the company is taking this as clear evidence that the drug should move into further late-stage testing.

The DMD space has been particularly volatile over the last several years as a slew of competitors have battled to bring a disease-modifying drug to market for the rare pediatric disease. Heart-breakingly, most of these drugs have shown mixed results at best in the clinic and have left the FDA in the uncomfortable position of having to tell patient advocacy groups full of desperate parents that these treatments just don't meet the standards for approval.

Highly regulated

The FDA surprised the masses this week with an unexpected approval of a generic for the multiple sclerosis drug Copaxone. Mylan N.V. has been working tirelessly for years to bring the 40 mg dose of the drug to market, but has been thwarted multiple times by both regulators and maker of the reference product Teva Pharmaceutical Industries Ltd.

Copaxone is by far Teva's best-selling drug and the approval puts nearly $4 billion in revenue in jeopardy for the already struggling company. Analysts had not anticipated an approval of the generic until 2018, if not even later.

The decision comes on the heels of new FDA Commissioner Scott Gottlieb taking a stance on the approval process for coplex generics —like Copaxone —and noting that there is going to be a new, easier regulatory pathway to help these products reach the market.